Unregulated gene therapy for longevity to launch

An anti-ageing gene therapy not approved by the US Food and Drug Administration (FDA) is set to be offered by an American company at overseas clinics outside of US jurisdiction.

The treatment, developed by Minicircle from Austin, Texas, uses a nonviral plasmid – a circular DNA sequence that does not change the person's genome – to instruct the body to produce increased levels of klotho, a protein vital in ageing and neurodegeneration. The company has opened a waitlist, and the therapy is set to be available this year to those willing to travel to Honduras, the Bahamas or Panama. It is yet to be tested in rigorous clinical trials, and has not been approved by the FDA or other major regulators.

'If you've got this thing in your body that is continuously producing a protein, maybe in five years' time you'll start to see serious adverse effects,' said Dr Christopher Gyngell, principal research fellow in biomedical ethics at the Department of Paediatrics, University of Melbourne, Australia to the New Scientist. 'People have died in trials of other gene therapies, even rigorously controlled ones with independent oversight, because it's still a novel area with a high risk of adverse effects.'

Early research on klotho has shown that mice lacking the protein would age rapidly and die young, while those genetically modified to produce excess klotho live up to 30 percent longer. In older monkeys, klotho injections have been found to improve memory. Furthermore, the levels of klotho in the body naturally decline with age in humans, which has motivated Minicircle to find a therapy to replenish it. Nevertheless, a case report of a child with increased levels of klotho due to a rare genetic disorder found she had weak bones and growth problems, indicating that too much protein could be damaging.

Minicircle is conducting a Phase I clinical trial to determine safety and toxicity in approximately 24 people at a site outside of the USA, which is not under FDA jurisdiction. The therapy, which is injected into a person's abdominal fat, is estimated to have an effect for up to a year.

Mac Davis, Minicircle founder and CEO, told the New Republic last year that he had some dizziness episodes and altered perception of time after his klotho gene therapy injection. He also stated that his food sensitivities were reduced, and his immune system felt stronger. However, the company has not published the clinical trial results yet, with its website stating that the data is being prepared for publication.

'The regulatory rules these companies [like Minicircle] complain of are not the red tape of overzealous bureaucrats,' Dr Christopher Rudge, sociolegal scholar and lecturer at Sydney Law School and deputy director of Sydney Health Law, Australia told the New Scientist. 'They're the direct inheritance of past tragedies, and that's easy to forget when you're focused only on the opportunities.'